Evidence map›Paper›PMID 39097643›Full record

Trial reportThe Journal of clinical endocrinology and metabolism2025

Adrenal Suppression From Vamorolone and Prednisone in Duchenne Muscular Dystrophy: Results From the Phase 2b Clinical Trial.

Alexandra Ahmet, Rebecca Tobin, Utkarsh J Dang, Raoul Rooman, Michela Guglieri, Paula R Clemens, Eric P Hoffman, Leanne M Ward

Abstract readRandomized Controlled TrialClinical Trial, Phase II
PubMed Full text at PMC Publisher
In one paragraph

Trial report in The Journal of clinical endocrinology and metabolism, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.

0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

16 citing papers in PubMed.

  1. Trial
  2. Trial
  3. Trial
  4. Trial
  5. Review
  6. Selective Glucocorticoid Receptor Modulators (SEGRAMs): Can They Improve the Therapeutic Index of Steroid Therapy?Medical science monitor : international medical journal of experimental and clinical research · 2026
    Review
  7. Article
  8. Article
  9. Review
  10. Review
  11. Article
  12. Review
  13. Article
  14. Article
  15. [Research progress on the pathogenesis and treatment strategies of Duchenne muscular dystrophy].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics
    Review
  16. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors.

Alexandra AhmetDivision of Endocrinology, Children's Hospital of Eastern Ontario and Department of Pediatrics, University of Ottawa, Ottawa, Ontario K1H 8L1, Canada.ORCID 0000-0002-3226-8322
Rebecca TobinDepartment of Health Sciences, Carleton University, Ottawa, Ontario K1S 5B6, Canada.
Utkarsh J DangDepartment of Health Sciences, Carleton University, Ottawa, Ontario K1S 5B6, Canada.
Raoul RoomanPendoCon bv, Putte 2580, Belgium.
Michela GuglieriJohn Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospital National Health Service Foundation Trust, Newcastle NE1 3BZ, UK.
Paula R ClemensDepartment of Neurology, University of Pittsburgh School of Medicine, Pittsburgh, PA 15213, USA.
Eric P HoffmanDepartment of Pharmaceutical Sciences, Binghamton University-State University of New York, Binghamton, New York 13790, USA.
Leanne M WardDivision of Endocrinology, Children's Hospital of Eastern Ontario and Department of Pediatrics, University of Ottawa, Ottawa, Ontario K1H 8L1, Canada.ORCID 0000-0003-1557-9185

Funding

Pivotal trial of vamorolone in Duchenne muscular dystrophyR44NS095423 · NINDS · REVERAGEN BIOPHARMA, INC. · PI CLEMENS, PAULA R, HOFFMAN, ERIC P. · 2016 to 2019
$6.1M
Dissociation of efficacy from side effects of anti-inflammatory therapies in Duchenne muscular dystrophyU54HD090254 · NICHD · CHILDREN'S RESEARCH INSTITUTE · PI CONKLIN, LAURIE S · 2016 to 2018
$2.5M
VBP15, an Innovative Steroid-like Intervention on DMD: VISION-DMDU34AR068616 · NIAMS · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI CLEMENS, PAULA R · 2015 to 2015
$300k
European Commission Horizons 2020 667078Institute of Arthritis and Musculoskeletal and Skin Diseases U34AR068616NationalNIAMS NIH HHS U34 AR068616NICHD NIH HHS U54 HD090254NIH HHSNINDS NIH HHS R44 NS095423NINDS NIH HHS R44NS095423
6 · The paper itself

Abstract

contextVamorolone, a novel "dissociative" steroid, demonstrated similar efficacy in muscle function relative to prednisone 0.75 mg/kg/day but improved linear growth and bone turnover markers in a randomized trial of pediatric Duchenne muscular dystrophy (DMD).

objectivesTo determine the frequency of adrenal suppression (AS) induced by vamorolone and prednisone in pediatric DMD and to assess cortisol thresholds using a monoclonal antibody immunoassay.

methodsPost hoc analysis of cortisol levels was performed on data from a randomized, double-blind, placebo- and prednisone-controlled 24-week trial of vamorolone with a 24-week crossover extension. Morning and ACTH-stimulated cortisol levels were measured using the Elecsys II immunoassay, with AS defined as a stimulated cortisol of <500 nmol/L ("historical threshold") and <400 nmol/L ("revised threshold").

resultsMean age at enrolment was 5.41 ± 0.86 years (n = 118). At week 24, the proportion of participants with AS using the historical and revised cortisol thresholds, respectively, were as follows: prednisone 0.75 mg/kg/day = 100% (25/25) and 92.0% (23/25); vamorolone 6 mg/kg/day = 95.2% (20/21) and 90.5% (19/21); vamorolone 2 mg/kg/day = 84.2% (16/19) and 47.5% (9/19); and placebo = 20.0% (4/20) and 0% (0/20). Morning and peak ACTH-stimulated cortisol were strongly correlated in steroid-treated boys (Spearman correlation week 48 = 0.83).

conclusionAS after vamorolone and prednisone was frequent and vamorolone-associated AS appeared dose-dependent. A lower stimulated cortisol threshold may be appropriate when using a monoclonal assay. We recommend hydrocortisone for glucocorticoid stress dosing in patients receiving vamorolone.

Indexed as

Adrenal InsufficiencyMuscular Dystrophy, DuchennePrednisonePregnadienediolsChildChild, PreschoolCross-Over StudiesDouble-Blind MethodGlucocorticoidsHumansHydrocortisoneMaleGlucocorticoidsHydrocortisonePrednisonePregnadienediolsVBP15 compoundadrenal insufficiencyadrenal suppressionDuchenne muscular dystrophyprednisonevamorolone

Identifiers

PMID39097643
PMCPMC11747748

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.